Tag Archives: Fyodor Urnov

CRISPR trials: the 2024 update

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The recent approval of Casgevy represents the first official success of gene editing-based therapies. The treatment for sickle cell anemia and thalassemia came in record time, only 11 years after CRISPR was invented. “Two diseases down, 5,000 to go,” commented Fyodor Urnov, Director of Technology & Translation at the Innovative Genomics Institute. Among the many diseases awaiting a cure, what will be the next to benefit from CRISPR? At what rate can we expect new treatments to arrive? The periodic update published by IGI is a must-read to navigate through hope and hype, papers and press-releases. The picture is overwhelmingly positive, but there is also some cause for disappointment. Here is an excerpt from the introduction:

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Patient-pioneer in the pantheon of medicine

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According to Fyodor Urnov, she should be added to “the pantheon of names inscribed in golden letters in the history of biomedicine.” That list includes other pioneering patients such as James Phipps (the boy vaccinated by Edward Jenner), Albert Alexander (the first human treated with penicillin), Louise Brown (the first test tube baby) and Emily Whitehead (the first recipient of CAR-T cells). Now the CRISPR Journal made the unusual decision to put her on the cover.

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Chronicles from the London editing summit

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CRISPR patient Victoria Gray talking at the summit (credit The Royal Society)

The third – and perhaps final – act of the Human Genome Editing Initiative ended last week. The first summit (Washington 2015) was held amid enthusiasm for the invention of CRISPR, with the aim of fostering a constructive dialogue between science and society. The second edition (Hong Kong 2018) was dominated by the birth in China of the first edited human beings. The main points in the agenda of geneticists and bioethicists meeting a few days ago (London 2023) was to overcome the shock and focus on the next challenges: broadening the range of treatable diseases, reducing the costs of therapies, simplifying them so they can be administered anywhere in the world, and reach as many sick people as possible.

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Just ignore He Jiankui, don’t feed his ego

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(Illustration by Mike McQuade, source Nature)

The Chinese scientist who edited the CRISPR babies was released from prison last spring. He tweets lightheartedly announcing that he has opened a new lab in Beijing. He claims to be dedicated to rare diseases. He is looking for funding that hopefully no one wants to give him. In the rogue experiment that made him famous, he violated so many ethical principles that the only thing one can hope for is that he changes jobs. Is it appropriate for influential newspapers and prestigious institutions to give him a limelight for this attempt to come back on the scene?

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Ode to Darwin, from Phages to Borgs

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Phages first, Borgs then. Jennifer Doudna and Jill Banfield published surprising new findings in Cell, suggesting that thousands of phages have stolen CRISPR from bacteria to deploy it against rivals. “CRISPR is so popular even viruses may use it,” Science jokes. Nature puts it seriously “CRISPR tools found in thousands of viruses could boost gene editing.”

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Fyodor Urnov imagines CRISPR cures

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Fyodor Urnov, University of California, Berkeley/Innovative Genomics Institute. Urnov is a pioneer in the field of genome editing, with a diverse background in academia, industry and the nonprofit sector. During his time at Sangamo Biosciences he co-developed and co-named human genome editing.

“Imagine CRISPR cures” is the title of the keynote by Fyodor Urnov at the World CRISPR Day conference on October 20. The talk was far from a celebration: “The fact that editing represents an approach to the majority of monogenic diseases in principle doesn’t mean that some biotech will take on disease number 823 in practice, and there are over 5,000 monogenic conditions on OMIM. Three years to IND in the best case scenario and cost scale of more than $6m per disease, that’s incompatible with either the promise of CRISPR to edit any given mutation which it can do or the unmet medical needs”. Don’t miss the on-demand video to learn more about the challenge of N=1 trials and Urnov’s call to arm against ultra-rare diseases.

CRISPR landmark trial: who said what?

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Credit Intellia Therapeutics

Here you can read a selection of notable comments about the landmark paper on in vivo genome-editing published in the New England Journal of Medicine on 26 June. The trial, conducted in the UK and New Zealand, produced the first-ever clinical data supporting the safety and efficacy of intravenous infusion of a single-dose CRISPR treatment. The treatment, developed by two US-based companies (Intellia Therapeutics and Regeneron Pharmaceuticals) targets a rare and fatal condition called transthyretin amyloidosis.

Jennifer Doudna (CRISPR co-inventor and co-founder of Intellia): “It’s a critical first step in being able to inactivate, repair, or replace any gene that causes disease, anywhere in the body” (source Science).

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Uninformed pontification on IQ editing

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According to his Twitter bio, Charles Murray is a “Husband, father, social scientist, writer, Madisonian. Or maybe right-wing ideologue, pseudoscientist, evil. Opinions differ.” You may remember him as the co-author of the controversial book “The Bell Curve” (1994), discussing purported connections between race and intelligence. The bad news is that he recently joined the CRISPR debate by tweeting “Gene editing to raise IQ will have a huge market”. The good news is that confutation is easy and a little irony is the best reply (check out Fyodor Urnov’s tweet in the gallery below).

Crispy weekend reads

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This is an issue for all tastes and interests. Don’t miss (Broken) Promises of Sustainable Food and Agriculture through New Biotechnologies by Todd Kuiken, Rodolphe Barrangou and Khara Grieger; A Code of Ethics for Gene Drive Research by George Annas and other members of the Controlling and Countering Gene Editing in Mosquitoes research project funded by the DARPA Safe Genes program; The Cas9 Hammer and the Sickle by Fyodor Urnov.

CRISPR targeting COVID-19

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Alexandra East-Seletsky graphic

Hopefully, CRISPR-based diagnostics will make an early debut amid COVID-19 outbreak. But what about a CRISPR prophylactic strategy to combat coronaviruses? The proof of concept is here, in bioRxiv, but it will be deployed in the next pandemic if we are lucky. It’s called PAC-MAN, like the videogame, stands for Prophylactic Antiviral CRISPR in huMAN cells, and comes from the Stanley Qi Lab.

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